Evidence map›Paper›PMID 41120126›Full record

Trial reportJournal for immunotherapy of cancer2025

Multicenter phase II trial of ipilimumab and nivolumab in metastatic or unresectable perivascular epithelioid cell tumor (PEComa): a substudy of

Sandip P Patel, Megan Othus, Young K Chae, Tali Azenkot, Michael J Wagner, Sara Threlkel, John H Farley, Christine M Magner, Helen X Chen, Elad Sharon and 3 more

Abstract readClinical Trial, Phase IIMulticenter Study
In one paragraph

Trial report in Journal for immunotherapy of cancer, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Review
  2. How I treat soft tissue sarcomas.Future oncology (London, England) · 2026
    Review
  3. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors.

Sandip P Patel *UC San Diego Health Moores Cancer Center, La Jolla, California, USA spatel@ucsd.edu.ORCID http://orcid.org/0000-0002-8387-4840
Megan OthusSWOG Statistical and Data Management Center, Fred Hutchinson Cancer Center, Seattle, Washington, USA.ORCID http://orcid.org/0000-0001-8176-6371
Young K Chae *Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
Tali AzenkotUniversity of California San Diego, La Jolla, California, USA.ORCID http://orcid.org/0000-0003-3329-6381
Michael J WagnerDana-Farber/Harvard Cancer Center, Boston, Massachusetts, USA.
Sara ThrelkelFred Hutchinson Cancer Research Center, Seattle, Washington, USA.
John H FarleyCancer Center at St. Joseph's, Dignity Health Arizona, Phoenix, Arizona, USA.
Christine M MagnerData Operations Center, SWOG, San Antonio, Texas, USA.
Helen X ChenCancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland, USA.
Elad SharonNational Cancer Institute, Bethesda, Maryland, USA.
Christopher W RyanOHSU, Portland, Oregon, USA.
Charles BlankeSWOG Network Operations Center, OHSU, Portland, Oregon, USA.
Razelle Kurzrock *Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

Funding

Member Site CoreU10CA180821 · NCI · BRIGHAM AND WOMEN'S HOSPITAL · PI Evanthia Galanis · 2014 to 2026
$177.3M
SWOG Statistics & Data Management Center complex - extension supplement for GY06U10CA180819 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI MEGAN OTHUS · 2014 to 2026
$115.2M
Northwestern University Lead Academic Participating Site Supplement Year 2UG1CA233320 · NCI · NORTHWESTERN UNIVERSITY AT CHICAGO · PI BENSON, AL B, MATEI, DANIELA E · 2019 to 2025
$5.5M
Hutchinson Center as Lead Academic Participating Site (U10)U10CA180828 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI APPELBAUM, FREDERICK · 2014 to 2018
$3.0M
NCI NIH HHS U10 CA180819NCI NIH HHS U10 CA180821NCI NIH HHS U10 CA180828NCI NIH HHS UG1 CA233320
6 · The paper itself

Abstract

backgroundMalignant perivascular epithelioid cell tumors (PEComas) are ultra-rare, aggressive mesenchymal neoplasms associated with poor outcomes. Standard-of-care systemic therapy includes mammalian target of rapamycin (mTOR) inhibition and chemotherapy. Immune checkpoint inhibition has not been previously studied in patients with advanced PEComas in a prospective clinical trial.

methodsThis is an open-label phase 2 trial of ipilimumab (1 mg/kg intravenously every 6 weeks) plus nivolumab (240 mg intravenously every 2 weeks) in rare tumors; here we report the cohort of patients with advanced malignant PEComa recruited from >1,000 sites across the USA. Primary end point was objective response rate, with progression-free survival, overall survival, and toxicity as secondary end points.

results17 patients were enrolled (N=16 evaluable). Median age was 59.5 years (range, 22-77 years) and the majority were female (81%). Best responses included partial response (18.8%), stable disease for <6 months (25.0%), and progressive disease (56.3%). Median OS was 7.5 months (95% CI 3.4 to 34.6) and median PFS was 1.9 months (95% CI 1.8 to 11.8). PFS in responding patients was 11.8, 18.9, and 34.6 months. Available genomic sequencing showed

conclusionsThe combination of ipilimumab and nivolumab demonstrated responses in 18.8% of patients with advanced malignant PEComas. Dual immunotherapy may be an alternative treatment option for certain patients, including those who are unable to tolerate or do not desire prolonged treatment with chemotherapy or mTOR inhibition or have failed to respond to other therapies. Ipilimumab and nivolumab warrants further investigation in this and other rare soft tissue sarcomas.

Indexed as

Antineoplastic Combined Chemotherapy ProtocolsCTLA-4 AntigenImmune Checkpoint InhibitorsIpilimumabNivolumabPerivascular Epithelioid Cell NeoplasmsProgrammed Cell Death 1 ReceptorAdultAgedFemaleHumansMaleMiddle AgedYoung AdultCTLA-4 AntigenCTLA4 protein, humanImmune Checkpoint InhibitorsIpilimumabNivolumabPDCD1 protein, humanProgrammed Cell Death 1 ReceptorImmune Checkpoint InhibitorIpilimumabNivolumabSolid tumor

Identifiers

PMID41120126
PMCPMC12542749

What OpenQuestion holds

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LicenceCC BY-NC
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.